Trientine Tetrahydrochloride Administered Once a Day for the First Line Treatment of Wilson's Disease Patients.
Orphalan
Summary
The goal of this clinical trial is to learn if a new trientine tetrahydrochloride (TETA 4HCl) formulation administered once a day compared to d-Penicillamine (DPA) as a first line treatment for people living with Wilson's disease (WD) is effective and safe. The study is enrolling children aged 8 years and older weighing at least 55 lb (25 kg) and adults with a recent diagnosis of WD. People recently diagnosed with WD, may be eligible for the study if they have either not started copper chelating treatment (such as DPA or trientine) or have been taking zinc salts for less than 28 days. Participants will be randomly allocated (like tossing a coin) to receive either DPA or TETA 4HCL for 48 weeks. During this time period participants will have up to 12 visits for health checks and assessments including blood and urine testing. In addition, at some visits participants may be asked to complete questionnaires on treatment satisfaction, and overall well-being.
Description
Wilson's disease (WD) is a rare, autosomal recessive genetic disorder of copper metabolism leading to progressive copper accumulation primarily in the liver and brain. Chelators are drugs that bind and remove copper from the body in the urine. d-Penicillamine (DPA) is currently the only approved first line chelator for the treatment of WD with trientine, an alternative copper chelator, only indicated for second line use. DPA is associated with numerous side effects which may lead to drug discontinuation in approximately 30% of people living with WD. Trientine is used following intolerance to D…
Eligibility
- Age range
- 8+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Participant is aged 8 years or older and is willing and able to give informed consent for participation in the study, or by a parent/legally authorized representative (LAR) and assent obtained (in accordance with local regulations) for any participant less than the age of majority (e.g. less than 18 years of age, depending on local requirements). 2. Participant has a body weight of at least 25 kg at screening. 3. Participant has a diagnosis of WD, as defined by a Leipzig score of greater than or equal to 4. Note that historical test results can be used for the diagnosis…
Interventions
- DrugTETA 4HCl formulation
The new formulation of TETA 4HCl will be administered once a day. Each film-coated tablet is scored to enable halving, if required. Randomized participants are planned to receive TETA 4HCl for the 48-week post-randomization period.
- DrugD-Penicillamine
Standard of care DPA is to be used, per the sites and treating physician's usual practice. To be administered in accordance with the product labelling and/or the institutions treatment practice guidelines. Randomised participants are planned to receive DPA for the 48-week post-randomization period.
Locations (3)
- University of Colorado Anschutz School of MedicineDenver, Colorado
- Yale University Medical CentreNew Haven, Connecticut
- University of Michigan Medical CentreAnn Arbor, Michigan